Hepatic Fibrosis, Congenital

Top-ranked pediatric hospital system with the lowest waitlist times marks historic milestone AURORA, Colo., Jan. 8, 2025 /PRNewswire/ -- Children's Hospital Colorado (Children's Colorado) has reached a historic milestone for its Liver Transplant Program, performing a record-breaking number of transplants in 2024. With 22 liver transplants completed, Children's Colorado surpassed the program's previous record of 20 transplants in 2021. As a nationally recognized transplant program, Children's Colorado has the only pediatric-focused, multi-specialty program in the Rocky Mountain region with the lowest waitlist times in the nation. It is one of only six pediatric transplant programs nationally with significant experience performing living donor liver transplants. This achievement reflects not only the steady growth of the Liver Transplant Program over the last decade but also the team's commitment to utilizing every available option possible to get children transplanted quickly, including grafts from directed living donors (donating to a specific person), non-directed living donors (donating to a stranger) and reduced-size or split grafts from deceased donors. "When I look at this data, it's striking to see that while living donors make up roughly 15% of pediatric liver transplants nationally, they account for 40% of transplants at Children's Colorado," said Dr. Amy Feldman, Medical Director of the Liver Transplant Program at Children's Colorado. "This highlights our unique expertise and experience in performing living donor liver transplants. With the help of our adult colleagues at the University of Colorado and the altruistic community, we are able to get kids transplanted before they experience significant morbidity and mortality." In 2024, patients traveling from out of state made up 43% of liver transplants performed at Children's Colorado, underscoring the program's regional and national impact. The breadth of diagnoses of children transplanted in 2024 – such as Wilson's disease, biliary atresia, acute liver failure, Budd-Chiari syndrome, congenital hepatic fibrosis, autosomal recessive polycystic kidney disease and non-cirrhotic portal hypertension – demonstrates the team's depth of expertise and ability to address complex medical needs quickly, before patients become too sick to transplant. "Achieving this milestone required tremendous dedication and teamwork," said Dr. Michael Wachs, Surgical Director of the Abdominal Transplant Program at Children's Colorado. "We have embraced cutting-edge advances, such as plasmapheresis and intraoperative dialysis for small children, allowing us to safely take on risks while maintaining excellent outcomes." The success of the Liver Transplant Program this past year would not have been possible without the steadfast support of Children's Colorado, UCHealth University of Colorado Hospital (UCHealth) and the unwavering commitment of the entire transplant team to deliver the lowest waitlist times in the country – less than two months, compared to the national average of eight months – and the best possible patient outcomes. As the program continues to grow, it is positioned to expand its reach and impact on a larger regional and national scale to help save the lives of more children. In partnership with UCHealth, any non-directed liver donor, or a person who donates a portion of their liver without knowing the person receiving the donation, will first be offered to a patient on the Children's Colorado waitlist. To be a living donor, one needs to be a healthy adult between the ages of 18-55. The donor does not need to be related to the recipient in any way to save their life. Surgeons at UCHealth are now able to perform the donor operation with robotic assistance, allowing for donors to be out of the hospital in just a few days. To learn more about the process of becoming a donor, go to UCHealth University of Colorado Hospital Transplant Center. For more information about pediatric liver transplants, visit Children's Colorado Liver Transplant Program. ABOUT CHILDREN'S HOSPITAL COLORADO Children's Hospital Colorado is one of the nation's leading and most expansive nonprofit pediatric healthcare systems with a mission to improve the health of children through patient care, education, research and advocacy. Founded in 1908 and ranked among the best children's hospitals in the nation as recognized by U.S. News & World Report, Children's Colorado has established itself as a pioneer in the discovery of innovative and groundbreaking treatments that are shaping the future of pediatric healthcare worldwide. Children's Colorado offers a full spectrum of family-centered care at its urgent, emergency and specialty care locations throughout Colorado, including an academic medical center on the Anschutz Medical Campus in Aurora, hospitals in Colorado Springs, Highlands Ranch and Broomfield, and outreach clinics across the region. For more information, visit or connect with us on Facebook, Instagram and YouTube. Children's Hospital Colorado complies with applicable Federal civil rights laws and does not discriminate on the basis of race, color, national origin, age, disability, or sex. ATENCIÓN: si habla español, tiene a su disposición servicios gratuitos de asistencia lingüística. Llame al 1-720-777-9800. CHÚ Ý: Nếu bạn nói Tiếng Việt, có các dịch vụ hỗ trợ ngôn ngữ miễn phí dành cho bạn. Gọi số 1-720-777-9800. Media Contacts: Blayke Roznowski, Children's Hospital Colorado 24/7 media line: 303-890-8314 [email protected] SOURCE Children's Hospital Colorado WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

128th Annual Boston Marathon is Monday, April 15th NEW YORK, April 12, 2024 /PRNewswire/ -- The American Liver Foundation (ALF) is proud to once again be a member of the Boston Marathon Official Charity Program for the 2024 Boston Marathon®, which takes place on Monday, April 15, 2024. As the Boston Athletic Association's first official charity team, ALF has a long history in Boston. Since 1988, the American Liver Foundation Marathon Team has made a dramatic impact on the American Liver Foundation's mission. Continue Reading American Liver Foundation (ALF) runners participate in the 2024 Boston Marathon® to raise awareness of liver disease! Post this "ALF is grateful for our history with the Boston Athletic Association, our partnership has been longstanding, and we are so appreciative," said Lorraine Stiehl, Chief Executive Officer, American Liver Foundation. "Our team is made up of many runners who have been profoundly affected by liver disease. As a runner myself, I'm inspired by their stories and dedication to their loved ones, and ALF." One of those running this year is Laura Yardumian. "I'm running this year in honor of my maternal grandfather who died of liver cancer, for myself after surviving liver cancer, and for all those currently living with this challenging disease," said Laura. This will be Laura's first time running the Boston Marathon®. As she starts in Hopkinton, and treks Heartbreak Hill, she will be joined by two special members of the ALF team, her sons Lorenzo Perez and Varandt Khodaverdian. "It is not so much about the time it takes to finish the race; it is about getting to the finish line in good health, educating people about liver disease during this five-month journey, and raising funds to support a very worthy organization." The American Liver Foundation team is made up of novice to experienced runners, most of whom have a connection to liver disease. Some of the 50-member team include: Molly Dillion: Running her first marathon for her dad who received his liver transplant in 2022. Corey Fugere: Running his first Boston Marathon for his eldest son, who was diagnosed with primary sclerosing cholangitis in 2022. Michael Kim: Celebrating his 50th birthday and 50 years of living with liver disease by running again with ALF. His last Boston Marathon® was in 2014. Fred and Kristin Kostecki: Joining Team ALF for the third year running for their daughter, who was diagnosed with congenital hepatic fibrosis with portal hypertension at age four. Read about the Kostecki family here: Sophie Long: Running again with ALF for her living liver donation recipient, Caitlin. Watch the video about their story here: Felix Lung: Running his first marathon with ALF in memory of his dad, who died of liver cancer. Jessica Moran: Running her first marathon in honor of her dad, who died of liver cancer. Gordon Scott: Running in honor of his two daughters who both have liver disease. He will be joined by his son-in-law, Steven Balla. Sarah and Emily Tully, Sydney Hsu: Running in honor of Keith Tully, Sarah and Emily's dad and "Coach Keith" to Sydney. After living with primary sclerosing cholangitis Keith received a liver transplant in July 2023. Learn more about ALF's 2024 Boston Marathon® team and support their 26.2 mile run at . About the American Liver Foundation American Liver Foundation (ALF) is a national community of patients, caregivers and medical professionals dedicated to helping people improve their liver health. Providing guidance and life-saving resources, we are a beacon for the 100 million Americans affected by liver disease. We advocate for patients and families, fund medical research and educate the public about liver wellness and disease prevention. We bring people together through our educational programs and events and create a network of support that lasts a lifetime. ALF is the largest organization focused on all liver diseases and the trusted voice for patients and families living with liver disease. For more information visit or call: 1 800 GO LIVER (800-465-4837). Contact: Julie Kimbrough [email protected] Direct dial: 646-737-9409 Facebook Twitter LinkedIn YouTube Instagram SOURCE American Liver Foundation

SAN DIEGO, May 11, 2021 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, today will give multiple oral and poster presentations at the American Society of Gene and Cell Therapy 2021 Virtual Annual Meeting being held May 11-14, 2021. The Company's oral presentation will highlight new data demonstrating the potential of its proprietary piggyBac DNA Delivery System for the treatment of genetic liver disorders in children and infants. Two additional presentations will highlight preclinical data supporting Poseida's first allogeneic CAR-T product candidate, P-BCMA-ALLO1 for R/R multiple myeloma, as well as preclinical data supporting the Company's anti-c-kit CAR-T program as a potentially safer preconditioning regimen for hematopoietic stem cell transplantation in patients with AML. "At Poseida, we are applying our proprietary technology platforms to develop the next wave of cell and gene therapies to not only treat severe cancers but to also unlock the potential of single treatment cures," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer. "The preclinical data being presented today further validate our CAR-T approach in multiple myeloma and in preconditioning for patients with AML, as well as demonstrate the exciting potential of our liver directed gene therapies, particularly in juvenile patients." Presentation Highlights: Oral Presentation: "Preclinical Evaluation of Combined Adeno-Associated Virus and Nanoparticle Delivery of piggyBac Transposon System for Durable Transgene Expression in the Growing Neonatal Murine Liver" Session Date/Time: Tuesday, May 11, 2021, 5:45pm - 6:00pm ET Abstract Number: 30 In a preclinical study, Poseida evaluated concomitant delivery of recombinant adeno-associated virus (rAAV) vectors and novel nanoparticle (NP) vectors using its piggyBac and "Super" piggyBac (SPB) technologies in order to deliver transposon and transposase in a growing neonatal mouse model. Data demonstrated that the piggyBac DNA Delivery System was effective in using both rAAV and NP vectors to introduce edited genes into targeted hepatocyte genomes. Poseida also found that SPB, a hyperactive form of the transposase, produced stable vector integration into the hepatocyte genome for more than three months, compared to transpose alone. Similarly, delivery of a novel NP formulation using SPB produced efficient delivery of mRNA to the liver hepatocytes, with similarly high levels of durability in the transgene expression. Taken together, these preclinical findings suggest the potential of piggyBac and SPB technology for gene therapies that treat congenital liver disease in infants and young children. Poster Presentation: "P-BCMA-ALL01: A Fully Allogeneic Stem Cell Memory T Cell (TSCM) CAR-T Therapy Targeting BCMA for the Treatment of Multiple Myeloma Shows Potent Anti-Tumor Activity" Session Date/Time: Tuesday, May 11, 2021, 8:00am – 10:00am ET Abstract Number: 789 P-BCMA-ALLO1 is Poseida's first fully allogenic product candidate targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. In in vitro and in vivo preclinical studies, P-BCMA-ALL01 showed effective, targeted cancer cell killing and cytokine secretion, with similar or superior performance in anti-tumor efficacy compared to an autologous CAR-T therapy. Inclusion of a proprietary "booster molecule" in the allogeneic manufacturing process further improved expansion of gene-edited cells and enabled production of hundreds of patient doses from a single manufacturing run, thereby reducing the manufacturing cost per dose into the same range as that of a monoclonal antibody. Poster Presentation: "Anti-c-kit CAR-T Cells Afford Effective Eradication of Human AML and Normal Hematopoietic Cells in a Preclinical Model of Safer Non-Genotoxic Stem Cell Transplant Conditioning" Session Date/Time: Tuesday, May 11, 2021, 8:00am – 10:00am ET Abstract Number: 715 Poseida is investigating its anti-c-kit CAR-T program, which leverages its proprietary piggyBac DNA Delivery System in preclinical studies as a potentially safer precursor conditioning therapy to the transplantation of hematopoietic stem cells (HSC) for patients suffering from AML. The piggyBac delivery vectors under investigation include a transposon that generates pure CAR+ product as well as a safety switch that allows rapid clearance of the reactive CAR-T cells prior to donor transplant of hematopoietic stem cells. Preclinical data to be presented in the poster showed that the lead CAR-T cells that express the anti-c-kit binder (CAR 1) deplete up to 92% of human CD34+ stem and progenitor cells in bone marrow within 48 hours. Additionally, an enhanced anti-c-kit CAR-T product, CAR 2, killed an estimated >99% of leukemia cells, exceeding the killing ability of a single dose of 30 mg/kg dose of busulfan. These encouraging data suggest that stem cell-directed CAR-T cells may be a safer preconditioning regimen compared to the current standard of care and may expand access to treatment for acute myeloid leukemia patients needing HSC transplant. About Poseida Therapeutics, Inc. Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary genetic engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-specific Gene Editing System and nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our wholly-owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics. To learn more, visit and connect with us on Twitter and LinkedIn. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding the clinical data presented, the potential benefits of Poseida's technology platforms and product candidates and Poseida's plans and strategy with respect to developing its technologies and product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon Poseida's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry, the fact that future clinical results could be inconsistent with results observed to date and the other risks described in Poseida's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Poseida undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Company Codes: NASDAQ-NMS:PSTX